Development and characterization of a fully humanized ACE2 mouse model

BMC Biol. 2025 Jul 1;23(1):194. doi: 10.1186/s12915-025-02293-w.

Abstract

Background: Many humanized angiotensin-converting enzyme 2 (ACE2) mouse models of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection do not replicate human ACE2 protein expression and thus exhibit pathology infrequently observed in humans. To address this limitation, we designed and characterized a fully humanized ACE2 (hACE2) mouse by replacing all exons/introns of the mouse Ace2 locus with human DNA comprising the entire ACE2 gene and an upstream long noncoding RNA (LncRNA).

Results: Compared to the popular Keratin18 ACE2 (KRT18-ACE2, K18) mouse model of SARS-CoV-2 infection, hACE2 mice displayed a similar tissue expression profile of ACE2 as that seen in human tissues. Further, hACE2 mice showed comparable blood pressure, angiotensin II metabolism, and renal cortical transcriptome as wild-type mice. Intranasal infection of K18 mice with the beta variant of SARS-CoV-2 resulted in high viral replication and inflammation of the lung and brain, weight loss, and compassionate euthanasia five days post-infection (PI). Similarly infected hACE2 mice displayed viral replication and inflammation in the lung (but not in brain), sustained weight, and 100% survival up to 12 days PI, with clear evidence of acquired immunity. CRISPR-mediated disruption of the upstream LncRNA caused minimal effects on ACE2 mRNA and protein.

Conclusions: The hACE2 model offers a more accurate approach to studying mechanisms underlying tissue-restricted expression of ACE2, elucidating noncoding sequence variants and an upstream LncRNA, and defining pathways relevant to human disease and associated co-morbidities.

Keywords: ACE2; Angiotensin; CRISPR; Long noncoding RNA; Mouse; SARS-CoV-2.

MeSH terms

  • Angiotensin-Converting Enzyme 2* / genetics
  • Angiotensin-Converting Enzyme 2* / metabolism
  • Animals
  • COVID-19* / genetics
  • COVID-19* / pathology
  • COVID-19* / virology
  • Disease Models, Animal*
  • Humans
  • Mice
  • Mice, Transgenic
  • RNA, Long Noncoding / genetics
  • SARS-CoV-2* / physiology

Substances

  • Angiotensin-Converting Enzyme 2
  • Ace2 protein, mouse
  • ACE2 protein, human
  • RNA, Long Noncoding